H.C. Wainwright Reiterates Buy Rating on Regenxbio Inc. (RGNX)

H.C. Wainwright analyst Yi Chen reiterated a Buy rating and $34.00 price target on Regenxbio Inc. (NASDAQ: RGNX).

The analyst commented: "Last Friday, REGENXBIO presented new 12-month data from the Phase 1/2/3 CAMPSIITE trial of clemidsogene lanparvovec (RGX-121) for the treatment of Mucopolysaccharidosis II (MPS II, aka Hunter syndrome) at the International Congress of Inborn Errors of Metabolism (ICIEM) 2025. In the pivotal phase (n=13), subjects sustained an 82% median reduction of cerebrospinal fluid (CSF) levels of heparan sulfate (HS) D2S6 through one year. The data are consistent with previously reported topline results from the CAMPSIITE trial, which met its primary endpoint of proportion of participants with CSF HS D2S6 below maximum attenuated levels at Week 16 with statistical significance (p<0.0001). Of note, pivotal subjects demonstrated neurodevelopmental skill acquisition or stability on all sub-scales of the Bayley Scales of Infant and Toddler Development, 3rd Edition (BSID-III) at one year, thus demonstrating a strong correlation between measured CSF HS D2S6 levels at Week 16 and neurocognitive outcomes at one year. This correlation supports the use of CSF HS D2S6 as a surrogate endpoint that is likely to predict clinical benefit under the accelerated approval pathway. REGENXBIO has submitted these longer-term pivotal data to the FDA for the ongoing Biologics License Application (BLA) review of RGX-121. In addition, the FDA completed a pre-license inspection and bioresearch monitoring information inspection for the RGX-121 BLA in August 2025 with no observations. In our view, the positive 12-month data should help RGX-121 secure U.S. regulatory approval in February 2026. We reiterate our Buy rating and $34 price target."

For an analyst ratings summary and ratings history on Regenxbio Inc. click here. For more ratings news on Regenxbio Inc. click here.

Shares of Regenxbio Inc. closed at $9.71 yesterday.

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